Aplastic anemia

This phase II trial is investigating whether eltrombopag, a drug that stimulates platelet production, can help children with severe aplastic anemia when combined with standard immunosuppressive therapy. The study will enroll children with newly diagnosed, relapsed, or refractory …

This trial is testing an experimental oral drug called PXS-5505 (amsulostat) in people with myelofibrosis, a type of bone marrow cancer. The study focuses on patients who are not eligible for a stem cell transplant. The main goal is to see if the drug is safe and tolerable, and t…

This study tests whether giving blood transfusions more liberally (when hemoglobin drops to 9 g/dL) versus more restrictively (when it drops to 7.5 g/dL) reduces death and serious complications like heart attack, stroke, or kidney injury in patients aged 70 and older undergoing n…

This Phase 3 trial tested a stem cell transplant using blood stem cells from a half-matched (haploidentical) donor in 21 people with various blood cancers. The goal was to see if this approach could help patients who don't have a fully matched donor. Participants received chemoth…

This phase 2 trial tests whether giving ruxolitinib (Jakafi) before, during, and after a stem cell transplant can improve outcomes for people with myelofibrosis. The study includes patients with intermediate-2 or high-risk disease, or intermediate-1 with additional risk factors. …

This study tested a drug called bitopertin in 15 adults with Diamond-Blackfan anemia (DBA), a rare inherited condition that causes severe anemia. Participants took the pill daily for 8 months, with doses possibly increasing over time. The goal was to see if the drug could raise h…

This study tested whether low doses of the hormone drug danazol can slow the shortening of telomeres—the protective caps on DNA—in people with telomere diseases. These conditions can cause bone marrow failure, lung scarring, and liver damage. Eighteen participants took danazol fo…

This study tested the drug RAD001 (everolimus) in 29 patients with relapsed or hard-to-treat blood cancers like leukemia, lymphoma, and myelofibrosis. The goal was to find the safest highest dose and see if it helps control the disease. It was a small early-phase trial at a singl…

This completed Phase 3 trial tested ravulizumab in 18 adults in China with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study measured changes in LDH, a marker of red blood cell breakdown, to see if the drug could control the…

This study tested the long-term safety of a drug called pegcetacoplan in people with PNH, a rare blood disease that destroys red blood cells. All 137 participants had already completed a prior pegcetacoplan study and continued treatment. The main goal was to track side effects ov…

This study tested a new drug called pelabresib in people with certain blood cancers, including myelofibrosis and leukemia. In the first part, the drug was given alone to find the safest dose. In the second part, it was given with or without another drug (ruxolitinib) to see if it…

This early-phase study tested a new drug called TQ05105 in 79 adults with certain blood cancers (myeloproliferative neoplasms). The main goals were to find the safest dose and understand side effects. The drug works by blocking a protein (JAK2) that can cause abnormal blood cell …

This study tested a new drug called TQ05105 in 107 people with moderate to high-risk myelofibrosis, a rare bone marrow disorder that often causes an enlarged spleen. The goal was to see if TQ05105 could shrink the spleen better than the standard drug hydroxyurea. Participants too…

This study tested eculizumab in 25 adults with PNH in China who had not previously taken complement inhibitors. Participants received infusions over 62 weeks. The main goal was to see if the drug reduced LDH levels, a marker of disease activity. Results showed a decrease in LDH, …

This early-phase study tested a new drug called TGR-1202 combined with the standard drug ruxolitinib in 60 people with myelofibrosis or polycythemia vera. The goal was to see if the combination is safe and tolerable. Participants either had not responded well to ruxolitinib alone…

This phase 2 trial tested a combination of rabbit antithymocyte globulin (Thymoglobuline) and ciclosporin in 35 people with acquired aplastic anemia, a condition where the bone marrow stops making enough blood cells. The goal was to see if this rabbit-based immune-suppressing tre…

This early study tested an experimental drug called OMS906 (zaltenibart) in 15 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease where red blood cells break apart. The main goal was to check safety and how the body handles the drug. Researchers also look…

This study followed 120 adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from the older drug eculizumab to the newer ravulizumab. Researchers measured changes in a blood marker called LDH to see if the new drug controlled red blood cell breakdown just as well or…

This study tested whether adding eltrombopag to standard immunosuppressive therapy improves early recovery in patients with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. 202 participants received either standard treatment (horse a…

This study tested the drug eltrombopag in 34 people with moderate aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. Eltrombopag is a pill that mimics a natural protein to stimulate blood cell production. Researchers measured whether it safely imp…

This study tested a two-step stem cell transplant for people with blood cancers like leukemia and lymphoma. First, patients received low-dose chemotherapy and radiation to prepare their bodies. Then, they got donor stem cells followed by drugs to prevent the donor cells from atta…

This study tested a combination of two drugs, fedratinib and nivolumab, in 30 adults with myelofibrosis whose disease no longer responded well to standard JAK-inhibitor treatment. The goal was to see if the combination could shrink the spleen, improve symptoms, or reduce the need…

This study tested a personalized geriatric optimization plan (GO!) in 30 adults aged 60 and older who were getting a donor stem cell transplant for blood cancers or bone marrow failure. The plan included tailored advice on diet, sleep, activity, and medications to improve physica…

This phase II trial tested whether adding the drug itacitinib to standard medications (tacrolimus and sirolimus) can better prevent graft-versus-host disease (GVHD) in 59 patients with blood cancers receiving donor stem cell transplants. GVHD occurs when donor immune cells attack…

This study tested a stem cell transplant from a half-matched family donor for people with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. The goal was to see if this approach could improve survival one year after the transplant. The tria…

This phase 2 trial looked at whether giving a JAK inhibitor (ruxolitinib) before a donor stem cell transplant helps people with myelofibrosis. The study enrolled 61 patients with primary or secondary myelofibrosis. The goal was to see if this approach improves survival and reduce…

This study tested a combination of two drugs, ruxolitinib and azacytidine, in 121 people with myelofibrosis or related blood cancers. The goal was to see if the combo could shrink tumors or improve blood counts. While the treatment aims to control the disease, it is not a cure, a…

This study tested whether adding the experimental drug navitoclax to the standard drug ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a type of bone marrow cancer. The main goal was to see if the combination could shrink an enlarged spleen by at le…

This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…

This pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…

This study reviewed medical records of 13 Korean children with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Researchers wanted to see if adding eltrombopag to standard immunosuppressive therapy was safe and effective. Over 6 months, t…

This study tested a new tablet called HSK39297 in 36 adults with paroxysmal nocturnal hemoglobinuria (PNH) who still had anemia despite stable treatment with anti-C5 antibodies. The goal was to see if the pill could raise hemoglobin levels and reduce the need for blood transfusio…

This study tested a new 9-day treatment plan for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. The treatment combines three drugs (rabbit antithymocyte globulin, cyclosporine, and levamisole) to suppress the immune sys…

This phase 2 trial tested a combination of two drugs, bortezomib and dexamethasone, in 18 adults with acquired pure red cell aplasia whose disease did not improve or came back after first treatment. The goal was to see if the combination could safely control the condition. The st…

This study tested a drug called navitoclax, either alone or added to the standard drug ruxolitinib, in 191 people with myelofibrosis. The main goal was to see if the treatment could shrink the spleen by at least 35% after 24 weeks. The trial also looked at whether it could reduce…

This study tested an oral medication called iptacopan in 52 adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who were already stable on standard infusion treatments. Participants switched from their usual therapy to iptacopan pills taken twice daily. The main goal was to see…

This study tested whether adding eltrombopag (a platelet booster) to standard immune-suppressing drugs (r-ATG and cyclosporine A) helps patients with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. Thirty-six East-Asian adults and c…

This phase 3 trial tested a new drug called HRS-5965 in 39 adults with paroxysmal nocturnal hemoglobinuria (PNH) who remained anemic even after standard anti-C5 antibody treatment. The study aimed to see if the drug could raise hemoglobin levels without needing blood transfusions…

This study tested whether a low dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (GVHD) — a common and serious complication where donor cells attack the patient's body. The trial involved 35 adults with various blood dis…

This study tested the drug dasatinib in 68 adults with several types of blood and bone marrow disorders, including certain leukemias, myelofibrosis, and mastocytosis. The goal was to see if dasatinib could help control the disease and to check its safety. Participants took dasati…

This study tested a drug called eltrombopag in 20 Chinese adults with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants had not responded to previous treatments. The drug was given daily for up to a year to see if it could imp…

This study tested a new pill called HSK39297 against a standard infusion drug (eculizumab) in 73 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. Participants had not received prior complement inhibitor treatment. The mai…

This study tested a two-step stem cell transplant for people with high-risk blood cancers like leukemia and lymphoma. First, patients got low-dose chemotherapy and radiation to prepare the body. Then, they received donor stem cells and immune cells to help destroy remaining cance…

This early-phase trial tested a new drug called flonoltinib maleate in 31 people with myeloproliferative neoplasms (MPNs), a group of bone marrow cancers. The main goals were to check safety, find the right dose, and see how the body processes the drug. Researchers hope this drug…

This study tested a new drug called OMS906 in 12 adults with paroxysmal nocturnal hemoglobinuria (PNH) who were not getting enough benefit from the standard treatment ravulizumab. The goal was to see if OMS906 is safe and can improve hemoglobin levels. Participants received OMS90…

This study reviews a modified transplant conditioning regimen for severe aplastic anemia in 72 patients across two hospitals. The approach uses a combination of fludarabine, melphalan, and cyclophosphamide to prepare the body for a stem cell transplant. Researchers looked at how …

This study looks at the long-term safety of bomedemstat, an oral drug, in 81 people with myeloproliferative neoplasms (MPNs) like essential thrombocythemia and primary myelofibrosis. Participants had already taken bomedemstat in a prior study. Researchers tracked side effects, sp…

This phase 2 trial tested whether adding luspatercept to standard cyclosporine treatment helps people with non-severe aplastic anemia. The study enrolled 58 newly diagnosed adults who had low blood counts but did not yet need transfusions. The goal was to see if the combination c…

This Phase 3 study tested a new oral medication, HRS-5965, against the standard infusion eculizumab in 76 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The main goal was to see if HRS-5965 could raise hemoglobin levels to normal without needing blo…

This phase 2 study tested the drug itacitinib in 4 people with myelofibrosis (a bone marrow disorder) who had already tried other treatments like ruxolitinib or fedratinib. The main goals were to check safety and find the right dose, and to see if it could shrink an enlarged sple…

This study tested whether quercetin, a natural substance found in some foods, could help prevent a type of mouth cancer in people with Fanconi anemia, a rare genetic condition that raises cancer risk. 48 participants took quercetin to see if it reduced certain cell changes linked…

This study looks back at medical records of people who had an allogeneic stem cell transplant to see whether romiplostim helped their platelets recover faster. Platelets are blood cells that help clotting, and low counts are common after transplant. The researchers will compare p…

This completed study looked at red and white blood cells in people with a rare blood disorder called PNH who also have bone marrow failure. Over 5,500 participants aged 10 and older with aplastic anemia or related conditions provided blood samples. The goal was simply to learn mo…

This completed study looked at blood vessel cells from 30 people with primary myelofibrosis, a rare bone marrow disorder. Researchers wanted to see if these cells act like stem cells that could be the origin of the disease. By growing the cells in the lab, they aimed to understan…

This study looked at how well two different capsule forms of TP-3654 are absorbed by the body and whether taking them with food changes absorption. It involved 24 healthy adults and was completed. The results will help design future studies for people with myelofibrosis, a bone m…

This study explored whether low hemoglobin (anemia) is linked to an enlarged heart (cardiomegaly) in children up to 18 years old. Researchers used chest X-rays and echocardiograms to check for heart enlargement in 56 anemic children. The goal was to better understand how anemia a…

This study looked at whether specially engineered versions of a natural immune protein (IL-2) can selectively boost regulatory T cells, which help calm the immune system. Researchers took blood samples from 67 people with various autoimmune or inflammatory conditions and tested t…

This study collected health information from over 6,000 people with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The goal was to track the safety of two medications, Soliris and Ultomiris, and to understand how PNH changes over time. Participants were followe…

This study reviewed medical records of 355 people in Turkey with myelofibrosis (a type of bone marrow cancer) who were treated with the drug ruxolitinib for at least 3 months. Researchers collected information on patient demographics, disease characteristics, and how the drug was…

This study looked at 70 people—some with primary myelofibrosis and some healthy—to understand how certain immune cells called MDSCs fuel inflammation in this rare bone marrow disease. Researchers also checked how these cells change with treatment and whether they help form new bl…

This study looked at 1,753 patients needing a stem cell transplant for blood cancers or other serious blood disorders. Researchers compared two strategies: first searching for a matched unrelated donor, then trying an alternative donor if needed, versus going straight to an alter…

This completed study followed 724 people in China with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers aimed to better understand how the disease progresses and how patients respond to different treatments, including the newly available drug eculizum…

This early-stage study tested a new drug, LP-005, in 68 healthy adults aged 18 to 50. The main goal was to check if the drug is safe and how the body handles it. This information will help design future studies for people with a rare blood disorder called PNH.

This phase 1 trial in 40 healthy adults tests how TQ05105, a drug for myelofibrosis, interacts with other medications that affect liver enzymes. Participants take TQ05105 alone and then with either itraconazole or rifampicin to see how these drugs change TQ05105 levels in the bod…

This completed registry study followed 80 children and young adults with severe immune cytopenias, including immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome. Researchers observed participants over up to 4 years to improve diagnosis and track the disease c…

This pilot study tested whether a combination of two drugs (G-CSF and plerixafor) could safely collect enough stem cells from the blood of children with Fanconi anemia for future gene therapy. Only 4 patients took part, and the main goal was to see if the cell collection process …